Hope in the face of no other options

When 4-year-old Phineas’s leukemia didn’t respond to conventional chemotherapy, his pediatric oncologist, Dr. Philip Roehrs, found him a clinical trial testing a groundbreaking new treatment called T-cell immunotherapy. Phineas, now 6, owes his life to that trial—and soon other North Carolina cancer patients will have access to T-cell immunotherapy trials at UNC.

In 2013, Carlos and Tina Sandi faced a fear that was all too familiar—the fear of losing a child to cancer.

Their 4-year-old son, Phineas, was diagnosed with acute lymphoblastic leukemia. Their daughter, Althea, had been diagnosed with a different form of leukemia six years earlier and died from the disease shortly after her second birthday.

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T-cell therapy clinical trials, like the one that saved 6-year-old Phineas’ life, will be offered at UNC beginning in 2016. (click to enlarge)
Facing a parent’s worst nightmare for a second time, the Sandi family sought treatment at UNC Hospitals. Within days of diagnosis, Phineas started aggressive chemotherapy under the care of pediatric oncologist Philip Roehrs, MD. Unfortunately, the treatments did not work.
 

“Even after several rounds of chemo, Phineas still had evidence of disease,” explains Dr. Roehrs, a member of the UNC Department of Pediatrics and UNC Lineberger Comprehensive Cancer Center. “In order for Phineas to get to the next stage in his care—to receive a bone marrow transplant—we needed him to be cancer-free, but the standard of care was just not working for him.”

With the chemotherapy ineffective, Dr. Roehrs began contacting centers testing a groundbreaking treatment called T-cell immunotherapy. The investigational therapy is designed to harness the power of the body’s own immune system to fight cancer with genetically reengineered T-cells that recognize and attack cancer cells.

“We had heard about these experimental trials that used a patient’s immune system to fight the cancer,” says Carlos. “The last thing we wanted to do was leave Chapel Hill and switch care teams, but at that point, it really was his only remaining option.”

Roehrs ultimately found a clinical trial that would enroll Phineas at the National Institutes of Health in Maryland. Phineas completed the NIH trial and was in full remission within 28 days, able to return to UNC for a life-saving bone marrow transplant.

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Dr. Philip Roehrs with the Sandi family. Left to right: Tina, Carlos, Phineas, Dr. Roehrs, and older sister, Fiona. (click to enlarge)
“The team that cared for Phineas is simply amazing,” reflects Tina. “There was a tremendous amount of communication between UNC and NIH throughout the trial, and Dr. Roehrs really advocated for our son throughout the entire process.”
 

Phineas was discharged from UNC on Christmas Day 2013 and today enjoys the activities of a normal 6 year old, most recently vacationing at Disney World and meeting new friends in kindergarten.

And because of amazing medical successes like that of Phineas, UNC will soon make T-cell immunotherapy available in North Carolina with the launch of a new clinical research program.

“What was once available in only a handful of cancer centers will now be available to patients in North Carolina,” says Jonathan Serody, MD, UNC Lineberger’s associate director of translational science.

UNC Lineberger has recruited two scientists from the Baylor College of Medicine, Barbara Savoldo, MD, PhD, and Gianpietro Dotti, MD, to help launch and lead T-cell therapy clinical trials. The pair has been studying these types of trials since 1998.

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Drs. Jon Serody, Gianpietro Dotti, and Barbara Savoldo (click to enlarge)
Beginning in early 2016, UNC Lineberger will open a trial for Hodgkin’s lymphoma. A trial for acute lymphoblastic leukemia (Phineas’ type of cancer) will follow next, with trials for other cancers, including solid tumors, being added as the program expands.
 

"It’s absolutely transformative,” says Carlos. “We are thrilled that immunotherapy is coming to North Carolina, just knowing what we do about the capabilities UNC has, about the wonderful people at UNC, about how much of an impact this institution has, and will continue to have, on the people of our state.”

The groundbreaking clinical trials will forever change the course of treatment for many families facing cancer.

“This will give people hope where there used to be no other options,” says Tina. “It could mean that their children get to live, that they will get to see their children grow up instead of planning their funerals. That’s what this really comes down to.”

More about T-cell therapy

T-cell therapy involves genetically re-engineering a type of the patient’s own disease-fighting white blood cells, called T-cells, to fight cancer. T-cells are taken from the patient’s blood and altered in the lab by inserting a gene, which reprograms them to recognize and attack cancerous cells. The altered cells are then reinfused into the patient’s body, where they disperse and multiply in order to identify and destroy rapidly growing tumor cells.

  • Immunotherapy harnesses the body’s own immune system to fight and defeat cancer with fewer side effects for the patient.
  • Immunotherapy is a powerful approach that systematically attacks cancer in the body.
  • Immunotherapy trains the immune system to target only the cancerous cells in the body.
  • Early clinical trials for patients with acute lymphoblastic leukemia (ALL) have shown more than 70% of patients with refractory leukemia responded to the treatment.